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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)PIK3CA-related Overgrowth Spectrum (PROS)RecruitingPhase 22026-10-09NCT06997588
A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic LeukemiaAbnormalities, Multiple, B-cell neoplasm, Burkitt lymphoma, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Virus Infections, Down syndrome, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Neurobehavioral Manifestations, Neurologic Manifestations, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Tumor Virus Infections, chromosomal disorder, hematologic disorder, hereditary disease, immune system disorder, immunoproliferative disorder, intellectual disability, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, neoplasm, nervous system disorder, viral infectious diseaseActive, not recruitingPhase 32026-10-01NCT03914625
A Study to Compare the Efficacy, Safety and Tolerability of FF/UMEC/VI With FF/VI in 12-17-year-olds With AsthmaHypersensitivity, Immediate, Respiratory Hypersensitivity, asthma, bronchial disorder, immune system disorder, lung disorder, obstructive lung disease, respiratory system disorderRecruitingPhase 32026-09-30NCT05757102
A Study to Find Out How Nerandomilast is Tolerated, Handled by the Body, and if it Helps Children and Adolescents With Interstitial Lung Disease (FIBRONEER-chILD)Fibrosing Interstitial Lung DiseaseRecruitingPhase 32026-09-29NCT07366034
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-28NCT07082725
A Study of Zanubrutinib Plus Anti-CD20 Versus Lenalidomide Plus Rituximab in Participants With Relapsed/Refractory Follicular or Marginal Zone LymphomaB-cell neoplasm, Hemic and Lymphatic Diseases, Lymphoma, B-Cell, Marginal Zone, Neoplasms by Histologic Type, follicular lymphoma, immune system disorder, immunoproliferative disorder, lymphatic system disorder, lymphoma, lymphoproliferative syndrome, marginal zone lymphoma, neoplasmActive, not recruitingPhase 32026-09-28NCT05100862
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 GangliosidosisBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Hemic and Lymphatic Diseases, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Niemann-Pick disease, Niemann-Pick disease type C, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, histiocytosis, inborn errors of metabolism, lymphatic system disorder, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, non-Langerhans cell histiocytosis, sphingolipidosisRecruitingPhase 32026-09-24NCT07054515
A Study of a New Way to Treat Children and Young Adults With a Brain Tumor Called NGGCTFemale Urogenital Diseases and Pregnancy Complications, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Pregnancy Complications, Neoplastic, Urogenital Diseases, adenocarcinoma, carcinoma, central nervous system nongerminomatous germ cell tumor, choriocarcinoma, embryonal carcinoma, epithelial neoplasm, malignant teratoma, mixed germ cell tumor, neoplasm, pineal region immature teratoma, pineal region yolk sac tumor, pregnancy disorder, teratoma, trophoblastic neoplasmRecruitingPhase 22026-09-23NCT04684368
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, GM1 gangliosidosis, GM2 gangliosidosis, Gangliosidoses, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosisActive, not recruitingPhase 32026-09-22NCT07082543
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3ARecruitingPhase 2 / Phase 32026-09-22NCT02716246
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPDBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorderActive, not recruitingPhase 32026-09-17NCT03911505
Aquarius Pilot Study to Evaluate the New Axonics Trial SystemDigestive System Diseases, Fecal Incontinence, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Lower Urinary Tract Symptoms, Male Urogenital Diseases, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Urinary Incontinence, Urinary Incontinence, Urge, Urination Disorders, Urogenital Diseases, Urological Manifestations, digestive system disorder, intestinal disorder, overactive bladder, rectal disorder, urinary bladder disorder, urinary system disorderRecruitingNot applicable2026-09-14NCT07335484
Cisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver Cancer After SurgeryNeoplasms by Histologic Type, fibrolamellar hepatocellular carcinoma, hepatoblastoma, mixed neoplasm, neoplasm, pediatric hepatocellular carcinomaActive, not recruitingPhase 32026-09-14NCT03533582
Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)Body Temperature Changes, Coma, Consciousness Disorders, Hypothermia, Hypoxia, Hypoxia, Brain, Neurobehavioral Manifestations, Neurologic Manifestations, Out-of-Hospital Cardiac Arrest, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Signs and Symptoms, Respiratory, Unconsciousness, brain disorder, brain hypoxia - ischemia, brain ischemia, cardiac arrest, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, heart disorder, nervous system disorder, vascular disorderRecruitingNot applicable2026-09-08NCT05376267
Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CPBronchiolitis Obliterans, Chronic Disease, Disease Attributes, Hemic and Lymphatic Diseases, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myeloproliferative Disorders, Neoplasms by Histologic Type, Pathologic Processes, Pathological Conditions, Signs and Symptoms, bone marrow disorder, bronchial disorder, bronchiolitis, bronchiolitis obliterans syndrome, bronchitis, cryptogenic organizing pneumonia, graft versus host disease, hematologic disorder, immune system disorder, leukemia, lung disorder, myeloid leukemia, neoplasm, obstructive lung disease, respiratory system disorderRecruitingPhase 22026-09-02NCT07354074
A Study of Lower Radiotherapy Dose to Treat Children With CNS GerminomaFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Urogenital Diseases, diabetes insipidus, endocrine system disorder, germinoma, kidney disorder, neoplasm, pineal region germinoma, pituitary gland disorder, urinary system disorderRecruitingPhase 22026-08-25NCT06368817
A Study of Combination Chemotherapy for Patients With Newly Diagnosed DAWT and Relapsed FHWTCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, Wilms tumor, benign urinary system neoplasm, hereditary disease, hereditary neoplastic syndrome, kidney cancer, kidney disorder, mixed neoplasm, neoplasm, urinary system disorderRecruitingPhase 22026-08-25NCT04322318
Inotuzumab Ozogamicin and Post-Induction Chemotherapy in Treating Patients With High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and B-LLyB-cell neoplasm, Burkitt lymphoma, DNA Virus Infections, Epstein-Barr virus infection, Hemic and Lymphatic Diseases, Herpesviridae infectious disease, Infections, Neoplasms by Histologic Type, Precursor B-Cell Lymphoblastic Leukemia-Lymphoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Tumor Virus Infections, acute biphenotypic leukemia, central nervous system leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoma, lymphoproliferative syndrome, mixed phenotype acute leukemia, neoplasm, testicular leukemia, viral infectious diseaseRecruitingPhase 32026-08-25NCT03959085
Project: Every Child for Younger Patients With CancerFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Langerhans cell histiocytosis specific to childhood, Male Urogenital Diseases, Myeloproliferative Disorders, Neoplasms by Histologic Type, Neoplasms by Site, Neoplasms, Connective and Soft Tissue, Neoplasms, Fibrous Tissue, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Skin and Connective Tissue Diseases, Urogenital Diseases, Urogenital Neoplasms, adrenal gland pheochromocytoma, benign urinary system neoplasm, bone marrow disorder, cancer, carcinoma, central nervous system cancer, childhood kidney neoplasm, congenital mesoblastic nephroma, connective tissue neoplasm, desmoid tumor, epithelial neoplasm, fibroma, hematologic disorder, immune system disorder, immunoproliferative disorder, in situ carcinoma, kidney cancer, kidney disorder, lymphatic system disorder, lymphoproliferative syndrome, melanocytic neoplasm, melanoma, mesoblastic nephroma, mixed neoplasm, myeloproliferative neoplasm, neoplasm, nervous system disorder, neuroendocrine neoplasm, neuroepithelial neoplasm, paraganglioma, pheochromocytoma, primitive neuroectodermal tumor, skin disorder, skin neoplasm, urinary system disorderRecruitingNot stated2026-08-25NCT02402244
Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk NeuroblastomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, Neuroectodermal Tumors, Primitive, Peripheral, epithelial neoplasm, ganglioneuroblastoma, neoplasm, neuroblastoma, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 32026-08-25NCT02176967
Minimising Adverse Drug Reactions and Verifying Economic Legitimacy in Children (MARVEL-PIC)neoplasmActive, not recruitingNot applicable2026-07-29NCT05667766
Combination Chemotherapy With or Without Bortezomib in Treating Younger Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or Stage II-IV T-Cell Lymphoblastic LymphomaHemic and Lymphatic Diseases, Neoplasms by Histologic Type, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, T-cell adult acute lymphocytic leukemia, T-cell childhood acute lymphocytic leukemia, hematologic disorder, immune system disorder, immunoproliferative disorder, leukemia, lymphatic system disorder, lymphoid leukemia, lymphoproliferative syndrome, neoplasmActive, not recruitingPhase 32026-07-29NCT02112916
Phase 2b Study of RPT904 as Monotherapy in Participants With IgE-Mediated Food AllergyEgg Hypersensitivity, Hypersensitivity, Immediate, Milk Hypersensitivity, Nut and Peanut Hypersensitivity, Peanut Hypersensitivity, food allergy, immune system disorderRecruitingPhase 22026-07-28NCT07220811
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3AEnrolling by invitationPhase 32026-07-17NCT04360265
Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven MedulloblastomaNeoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Neuroectodermal Tumors, Primitive, epithelial neoplasm, glioma, medulloblastoma, neoplasm, neuroepithelial neoplasm, primitive neuroectodermal tumorActive, not recruitingPhase 22026-07-16NCT02724579